Amyloidosis News and Research

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Amyloidosis is the term applied to a group of conditions caused by deposits of an abnormal protein called amyloid. This rare but serious condition affects between 1 and 5 people in every 100,000 each year in the UK. The condition is mainly seen among the elderly.

Advances in chemistry and delivery are taking oligonucleotide medicines beyond rare diseases

A review in Cell Reports Medicine examines how advances in oligonucleotide chemistry and delivery have established ASOs and RNA interference therapies as a class of genetic medicines, with 21 oligonucleotides approved at the time of writing. It also identifies extrahepatic delivery, safety, target validation, individualized treatment, and data quality as priorities as the field expands into common diseases and explores AI-assisted development.

24 Sep 2026

AI learned from cardiologists’ notes, and needed far less labeled ECG data

By teaching an AI system to connect raw heart signals with the clinical insights contained in cardiologists’ reports, researchers tested whether routine ECGs could reveal more with far fewer labeled examples.

2 Sep 2026

ECG-CLIP improves heart disease detection using less labeled data

Clinicians often use a medical test called a 12-lead electrocardiogram (ECG) to diagnose heart problems, which uses electrodes placed on the chest and limbs to record the heart's electrical activity.

1 Sep 2026

CARDIO-TTRansform trial investigating eplontersen fails to meet primary efficacy endpoint

The CARDIO-TTRansform trial investigating eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy did not meet its primary efficacy endpoint, according to results presented in a Hot Line session today at ESC Congress 2026.

28 Aug 2026

Single-dose gene therapy extends healthy lifespan in older mice

A research team from the Universitat Autònoma de Barcelona (UAB) has shown that a one-time administration of a gene therapy expressing the metabolic factor FGF21 can prolong health span in old mice.

15 Jun 2026

American Heart Association launches national heart transplant research network

Nearly 60 years after the first successful heart transplant, the American Heart Association, a relentless force changing the future of health for everyone everywhere, is launching a bold new initiative to fundamentally transform how heart transplant care is delivered across the United States - addressing long-standing gaps in innovation, equity and patient outcomes.

3 Jun 2026

Roswell Park scientists present five key cancer studies at clinical meeting

The research findings of experts from Roswell Park Comprehensive Cancer Center will be featured during the American Society of Clinical Oncology (ASCO) annual meeting May 29 to June 2 at McCormick Place in Chicago.

25 May 2026

New trial aims to improve ATTR-CM diagnosis in heart failure

Transthyretin amyloid cardiomyopathy (ATTR-CM) is an increasingly recognized yet frequently underdiagnosed cause of heart failure, particularly among older adults.

13 May 2026

First European trial tests CAR T therapy in amyloidosis patients

Three patients with light chain amyloidosis have been treated with CAR T cell therapy in the first European clinical trial testing the safety and efficacy of this immunotherapy in this patient group, led by UCL and UCLH researchers.

6 May 2026

Study links antimicrobial peptides to amyloid disease and neurodegeneration

For decades, antimicrobial peptides (AMPs) and amyloid-forming peptides were studied in largely separate contexts.

5 May 2026

Blood protein structure changes may enable earlier detection of Alzheimer’s

Advanced proteomics and AI reveal blood protein changes, offering insights into early Alzheimer's detection and differentiation from mild cognitive impairment.

13 Mar 2026

Blood marker linked to Alzheimer’s also found in systemic amyloidosis

A certain blood protein regarded as an early indicator of Alzheimer's disease also appears to play a role in other disorders.

11 Mar 2026

Natural compound from pomegranate leaves shows promise against TTR amyloidosis

A research team in Kumamoto University has discovered that a natural compound found in pomegranate leaves and branches can directly break down harmful protein aggregates linked to transthyretin (TTR) amyloidosis, a progressive and potentially life-threatening disease affecting the nerves and heart.

23 Feb 2026

Alzheimer’s plaques decline after CAR-T immune cell treatment in preclinical study

Researchers engineered CD4+ chimeric antigen receptor T cells targeting fibrillar amyloid beta and tested them in a mouse Alzheimer model. The preclinical study showed reduced amyloid pathology and altered neuroimmune responses, supporting CAR-T immunotherapy as a potential future Alzheimer strategy.

11 Feb 2026

Minimally invasive biopsy enables real-time tracking of amyloid disease progression

Transthyretin amyloidosis (ATTR) is a rare, progressive and highly aggressive degenerative disease. It is caused by the misfolding of a specific protein, leading to its toxic accumulation in the form of filamentous deposits in various organs.

26 Jan 2026

CRISPR-Cas3: A safer gene-editing tool shows promise for transthyretin amyloidosis treatment

Genetic disorders occur due to alterations in the primary genetic material, deoxyribonucleic acid (DNA), of an organism. Transthyretin amyloidosis (ATTR) is a progressive disorder involving amyloid deposits of misfolded transthyretin (TTR) proteins.

14 Jan 2026

Lysophosphatidylcholines show gene-dependent effects in early Alzheimer’s disease

A new study presents robust evidence on the role of lysophosphatidylcholines (LPCs) in the onset of Alzheimer's disease. Researchers discovered that LPCs-compounds that transport a variety of healthy fatty acids to the brain-either promote Alzheimer's or protect against it, depending on a person's genetics.

17 Dec 2025

Atrial dysfunction assessment tool may help identify stroke risk in people with ATTR-CM

A hidden atrial dysfunction may raise stroke risk in people with transthyretin amyloid cardiomyopathy (ATTR-CM); however, a non-invasive assessment tool may help identify those at increased risk, according to a preliminary study to be presented at the American Heart Association's Scientific Sessions 2025.

3 Nov 2025

Advances in diagnosis and management of systemic light chain amyloidosis

Systemic light chain (AL) amyloidosis is a rare and life-threatening disorder characterized by the deposition of misfolded immunoglobulin light chains as insoluble amyloid fibrils in various tissues and organs, leading to progressive structural and functional impairment.

30 Oct 2025

Gut disorders forecast Alzheimer’s and Parkinson’s risk years before diagnosis

Study maps clinical, genetic, and proteomic links between gut-brain disorders and neurodegeneration, showing how diabetes, vitamin D deficiency, and bowel disorders forecast Alzheimer’s and Parkinson’s years in advance. Multimodal models in biobanks highlight early detection and personalized prevention opportunities.

28 Aug 2025